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1. Genotype, oxidase status, and preceding infection or autoinflammation do not affect allogeneic HCT outcomes for CGD

Author

Leiding, Jennifer W., Arnold, Danielle E., Parikh, Suhag, Logan, Brent, Marsh, Rebecca A., Griffith, Linda M., Wu, Ruizhe, Kidd, Sharon, Mallhi, Kanwaldeep, Chellapandian, Deepak, Si Lim, Stephanie J., Grunebaum, Eyal, Falcone, E. Liana, Murguia-Favela, Luis, Grossman, Debbi, Prasad, Vinod K., Heimall, Jennifer R., Touzot, Fabien, Burroughs, Lauri M., Bleesing, Jack, Kapoor, Neena, Dara, Jasmeen, Williams, Olatundun, Kapadia, Malika, Oshrine, Benjamin R., Bednarski, Jeffrey J., Rayes, Ahmad, Chong, Hey, Cuvelier, Geoffrey D. E., Forbes Satter, Lisa R., Martinez, Caridad, Vander Lugt, Mark T., Yu, Lolie C., Chandrakasan, Shanmuganathan, Joshi, Avni, Prockop, Susan E., Dávila Saldaña, Blachy J., Aquino, Victor, Broglie, Larisa A., Ebens, Christen L., Madden, Lisa M., DeSantes, Kenneth, Milner, Jordan, Rangarajan, Hemalatha G., Shah, Ami J., Gillio, Alfred P., Knutsen, Alan P., Miller, Holly K., Moore, Theodore B., Graham, Pamela, Bauchat, Andrea, Bunin, Nancy J., Teira, Pierre, Petrovic, Aleksandra, Chandra, Sharat, Abdel-Azim, Hisham, Dorsey, Morna J., Birbrayer, Olga, Cowan, Morton J., Dvorak, Christopher C., Haddad, Elie, Kohn, Donald B., Notarangelo, Luigi D., Pai, Sung-Yun, Puck, Jennifer M., Pulsipher, Michael A., Torgerson, Troy R., Malech, Harry L., and Kang, Elizabeth M.

Published
2023
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2. Infections in Infants with SCID: Isolation, Infection Screening, and Prophylaxis in PIDTC Centers.

Author

Dorsey, Morna J, Wright, Nicola AM, Chaimowitz, Natalia S, Dávila Saldaña, Blachy J, Miller, Holly, Keller, Michael D, Thakar, Monica S, Shah, Ami J, Abu-Arja, Rolla, Andolina, Jeffrey, Aquino, Victor, Barnum, JL, Bednarski, Jeffrey J, Bhatia, Monica, Bonilla, Francisco A, Butte, Manish J, Bunin, Nancy J, Chandra, Sharat, Chaudhury, Sonali, Chen, Karin, Chong, Hey, Cuvelier, Geoffrey DE, Dalal, Jignesh, DeFelice, Magee L, DeSantes, Kenneth B, Forbes, Lisa R, Gillio, Alfred, Goldman, Fred, Joshi, Avni Y, Kapoor, Neena, Knutsen, Alan P, Kobrynski, Lisa, Lieberman, Jay A, Leiding, Jennifer W, Oshrine, Benjamin, Patel, Kiran P, Prockop, Susan, Quigg, Troy C, Quinones, Ralph, Schultz, Kirk R, Seroogy, Christine, Shyr, David, Siegel, Subhadra, Smith, Angela R, Torgerson, Troy R, Vander Lugt, Mark T, Yu, Lolie C, Cowan, Morton J, Buckley, Rebecca H, Dvorak, Christopher C, Griffith, Linda M, Haddad, Elie, Kohn, Donald B, Logan, Brent, Notarangelo, Luigi D, Pai, Sung-Yun, Puck, Jennifer, Pulsipher, Michael A, and Heimall, Jennifer

Subjects
Humans, Severe Combined Immunodeficiency, Disease Susceptibility, Neonatal Screening, Prognosis, Antibiotic Prophylaxis, Hematopoietic Stem Cell Transplantation, Age of Onset, Infection Control, Infant, Infant, Newborn, Disease Management, Female, Male, Public Health Surveillance, Time-to-Treatment, Surveys and Questionnaires, Clinical Decision-Making, Infections, hematopoietic stem cell transplant, newborn screening, primary immunodeficiency, prophylaxis, severe combined immunodeficiency, Stem Cell Research, Perinatal Period - Conditions Originating in Perinatal Period, Pediatric, Prevention, Regenerative Medicine, Transplantation, Infant Mortality, Health Services, Clinical Research, Infection, Immunology
Abstract

PurposeThe Primary Immune Deficiency Treatment Consortium (PIDTC) enrolled children with severe combined immunodeficiency (SCID) in a prospective natural history study of hematopoietic stem cell transplant (HSCT) outcomes over the last decade. Despite newborn screening (NBS) for SCID, infections occurred prior to HSCT. This study's objectives were to define the types and timing of infection prior to HSCT in patients diagnosed via NBS or by family history (FH) and to understand the breadth of strategies employed at PIDTC centers for infection prevention.MethodsWe analyzed retrospective data on infections and pre-transplant management in patients with SCID diagnosed by NBS and/or FH and treated with HSCT between 2010 and 2014. PIDTC centers were surveyed in 2018 to understand their practices and protocols for pre-HSCT management.ResultsInfections were more common in patients diagnosed via NBS (55%) versus those diagnosed via FH (19%) (p = 0.012). Outpatient versus inpatient management did not impact infections (47% vs 35%, respectively; p = 0.423). There was no consensus among PIDTC survey respondents as to the best setting (inpatient vs outpatient) for pre-HSCT management. While isolation practices varied, immunoglobulin replacement and antimicrobial prophylaxis were more uniformly implemented.ConclusionInfants with SCID diagnosed due to FH had lower rates of infection and proceeded to HSCT more quickly than did those diagnosed via NBS. Pre-HSCT management practices were highly variable between centers, although uses of prophylaxis and immunoglobulin support were more consistent. This study demonstrates a critical need for development of evidence-based guidelines for the pre-HSCT management of infants with SCID following an abnormal NBS.Trial registrationNCT01186913.

Published
2021

4. Higher Risks of Toxicity and Incomplete Recovery in 13- to 17-Year-Old Females after Marrow Donation: RDSafe Peds Results.

Author

Pulsipher, Michael A, Logan, Brent R, Kiefer, Deidre M, Chitphakdithai, Pintip, Riches, Marcie L, Rizzo, J Douglas, Anderlini, Paolo, Leitman, Susan F, Varni, James W, Kobusingye, Hati, Besser, RaeAnne M, Miller, John P, Drexler, Rebecca J, Abdel-Mageed, Aly, Ahmed, Ibrahim A, Ball, Edward D, Bolwell, Brian J, Bunin, Nancy J, Cheerva, Alexandra, Delgado, David C, Dvorak, Christopher C, Gillio, Alfred P, Hahn, Theresa E, Hale, Gregory A, Haight, Ann E, Hayes-Lattin, Brandon M, Kasow, Kimberly A, Linenberger, Michael, Magalhaes-Silverman, Margarida, Mori, Shahram, Prasad, Vinod K, Quigg, Troy C, Sahdev, Indira, Schriber, Jeffrey R, Shenoy, Shalini, Tse, William T, Yanik, Gregory A, Navarro, Willis H, Horowitz, Mary M, Confer, Dennis L, Shaw, Bronwen E, and Switzer, Galen E

Subjects
Humans, Pain, Tissue and Organ Harvesting, Bone Marrow Transplantation, Transplantation, Homologous, Age Factors, Sex Factors, Time Factors, Adolescent, Tissue Donors, Female, Male, BM collection toxicities, Donor safety, PBSC collection toxicities, Stem cell transplantation, Transplantation, Chronic Pain, Neurosciences, Pediatric, Clinical Research, Stem Cell Research, Pain Research, Clinical Trials and Supportive Activities, Prevention, Immunology, Clinical Sciences
Abstract

Although donation of bone marrow (BM) or peripheral blood stem cells (PBSCs) from children to family members undergoing allogeneic transplantation are well-established procedures, studies detailing levels of pain, symptoms, and long-term recovery are lacking. To address this lack, we prospectively enrolled 294 donors age 20% of females and males age 13 to 17 years do not return to baseline pain levels by 1 year after BM donation. Studies aimed at decreasing symptoms and improving recovery in older children are warranted.

Published
2019

5. Effect of Aging and Predonation Comorbidities on the Related Peripheral Blood Stem Cell Donor Experience: Report from the Related Donor Safety Study.

Author

Pulsipher, Michael A, Logan, Brent R, Chitphakdithai, Pintip, Kiefer, Deidre M, Riches, Marcie L, Rizzo, J Douglas, Anderlini, Paolo, Leitman, Susan F, Varni, James W, Kobusingye, Hati, Besser, RaeAnne M, Miller, John P, Drexler, Rebecca J, Abdel-Mageed, Aly, Ahmed, Ibrahim A, Akard, Luke P, Artz, Andrew S, Ball, Edward D, Bayer, Ruthee-Lu, Bigelow, Carolyn, Bolwell, Brian J, Broun, E Randolph, Bunin, Nancy J, Delgado, David C, Duckworth, Katharine, Dvorak, Christopher C, Hahn, Theresa E, Haight, Ann E, Hari, Parameswaran N, Hayes-Lattin, Brandon M, Jacobsohn, David A, Jakubowski, Ann A, Kasow, Kimberly A, Lazarus, Hillard M, Liesveld, Jane L, Linenberger, Michael, Litzow, Mark R, Longo, Walter, Magalhaes-Silverman, Margarida, McCarty, John M, McGuirk, Joseph P, Mori, Shahram, Prasad, Vinod K, Rowley, Scott D, Rybka, Witold B, Sahdev, Indira, Schriber, Jeffrey R, Selby, George B, Shaughnessy, Paul J, Shenoy, Shalini, Spitzer, Thomas, Tse, William T, Uberti, Joseph P, Vusirikala, Madhuri, Waller, Edmund K, Weisdorf, Daniel J, Yanik, Gregory A, Navarro, Willis H, Horowitz, Mary M, Switzer, Galen E, Shaw, Bronwen E, and Confer, Dennis L

Subjects
Humans, Peripheral Blood Stem Cell Transplantation, Comorbidity, Adolescent, Adult, Aged, Middle Aged, Blood Donors, Female, Male, Young Adult, Peripheral Blood Stem Cells, BM collection toxicities, Donor safety, PBSC collection toxicities, Stem cell transplantation, Transplantation, Pain Research, Stem Cell Research - Nonembryonic - Human, Clinical Research, Chronic Pain, Stem Cell Research, Aging, Clinical Sciences, Immunology
Abstract

The development of reduced-intensity approaches for allogeneic hematopoietic cell transplantation has resulted in growing numbers of older related donors (RDs) of peripheral blood stem cells (PBSCs). The effects of age on donation efficacy, toxicity, and long-term recovery in RDs are poorly understood. To address this we analyzed hematologic variables, pain, donation-related symptoms, and recovery in 1211 PBSC RDs aged 18 to 79 enrolled in the Related Donor Safety Study. RDs aged > 60 had a lower median CD34+ level before apheresis compared with younger RDs (age > 60, 59 × 106/L; age 41 to 60, 81 × 106/L; age 18 to 40, 121 × 106/L; P < .001). This resulted in older donors undergoing more apheresis procedures (49% versus 30% ≥ 2 collections, P < .001) and higher collection volumes (52% versus 32% > 24 L, P < .001), leading to high percentages of donors aged > 60 with postcollection thrombocytopenia

Published
2019

6. Reduced-Intensity/Reduced-Toxicity Conditioning Approaches Are Tolerated in XIAP Deficiency but Patients Fare Poorly with Acute GVHD

Author

Arnold, Danielle E., Nofal, Rofida, Wakefield, Connor, Lehmberg, Kai, Wustrau, Katharina, Albert, Michael H., Morris, Emma C., Heimall, Jennifer R., Bunin, Nancy J., Kumar, Ashish, Jordan, Michael B., Cole, Theresa, Choo, Sharon, Brettig, Tim, Speckmann, Carsten, Ehl, Stephan, Salamonowicz, Malgorzata, Wahlstrom, Justin, Rao, Kanchan, Booth, Claire, Worth, Austen, and Marsh, Rebecca A.

Published
2022
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7. Tabelecleucel for EBV+ PTLD following allogeneic HCT or SOT in a multicenter expanded access protocol

Author

Nikiforow, Sarah, primary, Whangbo, Jennifer S, additional, Reshef, Ran, additional, Tsai, Donald E, additional, Bunin, Nancy J., additional, Abu-Arja, Rolla F, additional, Mahadeo, Kris Michael, additional, Weng, Wen-Kai, additional, Van Besien, Koen, additional, Loeb, David, additional, Nasta, Sunita D., additional, Nemecek, Eneida R., additional, Zhao, Weizhi, additional, Sun, Yan, additional, Galderisi, Faith C., additional, Wahlstrom, Justin, additional, Mehta, Aditi, additional, Gamelin, Laurence Isabelle, additional, Dinavahi, Rajani, additional, and Prockop, Susan E., additional

Published
2024
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8. Partially CD3+-Depleted Unrelated and Haploidentical Donor Peripheral Stem Cell Transplantation Has Favorable Graft-versus-Host Disease and Survival Rates in Pediatric Hematologic Malignancy

Author

Seif, Alix E., Li, Yimei, Monos, Dimitri S., Heidemann, Stephanie C., Aplenc, Richard, Barrett, David M., Casper, James T., Freedman, Jason L., Grupp, Stephan A., Margolis, David A., Olson, Timothy S., Teachey, David T., Keever-Taylor, Carolyn A., Wang, Yongping, Talano, Julie-An M., and Bunin, Nancy J.

Published
2020
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9. Pilot Study of Unrelated Donor (URD) Peripheral Stem Cell Transplant (PSCT) with CD45RA-Depleted Addback Following TCRab/CD19 Depletion in Pediatric Patients with Hematologic Malignancies

Author

Elgarten, Caitlin W., primary, Heidemann, Stephanie, additional, Freedman, Jason L., additional, Grupp, Stephan A., additional, Hankins, Patricia, additional, Kadauke, Stephan, additional, Li, Yimei, additional, McGlynn, Barbara, additional, Olson, Timothy S., additional, Wang, Yongping, additional, Wray, Lisa, additional, Wang, Lei, additional, Seif, Alix E., additional, and Bunin, Nancy J., additional

Published
2024
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10. Outcomes Following Matched Sibling Donor Transplant for Severe Combined Immunodeficiency: A Report from the Pidtc

Author

Rayes, Ahmad, primary, Logan, Brent, additional, Liu, Xuerong, additional, Dara, Jasmeen, additional, Buckley, Rebecca H., additional, Oved, Joseph H., additional, Kapoor, Neena, additional, Kapadia, Malika, additional, Chandra, Sharat, additional, Martinez, Caridad, additional, Bunin, Nancy J., additional, Chandrakasan, Shanmuganathan, additional, Burroughs, Lauri M., additional, Bednarski, Jeffrey J, additional, Haines, Hilary, additional, Cuvelier, Geoff D.E., additional, Eissa, Hesham, additional, Talano, Julie A, additional, Saldana, Blachy J, additional, Ebens, Christen L., additional, Chaudhury, Sonali, additional, Shereck, Evan, additional, Aquino, Victor, additional, Knutsen, Alan P., additional, Alexander, Jessie L, additional, Gillio, Alfred P., additional, Chellapandian, Deepak, additional, Shah, Ami J., additional, Miller, Holly K., additional, Lugt, Mark T. Vander, additional, DeSantes, Kenneth, additional, Dorsey, Morna, additional, Parrott, Roberta E, additional, O'Reilly, Richard J., additional, Aguayo-Hiraldo, Paibel, additional, Torgerson, Troy, additional, Leiding, Jennifer W., additional, Marsh, Rebecca A., additional, Griffith, Linda M., additional, Pulsipher, Mike A., additional, Kohn, Donald B., additional, Notarangelo, Luigi D., additional, Cowan, Morton J., additional, Puck, Jennifer, additional, Heimall, Jennifer R., additional, Haddad, Elie, additional, Pai, Sung-Yun, additional, and Dvorak, Christopher C., additional

Published
2024
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11. Optimal Rabbit Antithymocyte Pharmacokinetics (PK) Is Associated with Improved Disease-Free Survival (DFS) in TCR-Αβ/CD19-Depleted Pediatric Haploidentical Hematopoietic Cell Transplantation for Hematologic Malignancies

Author

Dvorak, Christopher C., primary, Holbrook-Brown, Lucia, additional, Long-Boyle, Janel Renee, additional, Abdel-Azim, Hisham, additional, Bertaina, Alice, additional, Vatsayan, Anant, additional, Talano, Julie A, additional, Bunin, Nancy J., additional, Anderson, Eric J, additional, Flower, Allyson, additional, Lalefar, Nahal, additional, Higham, Christine S., additional, Kapoor, Neena, additional, Odinakachukwu, Maryanne A, additional, Jacobsohn, David A, additional, and Pulsipher, Mike A., additional

Published
2024
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12. Outcomes of Unrelated Partial T Cell Depleted Stem Cell Transplantation for Patients with Hemoglobinopathies

Author

Gibson, Nora M., primary, Elgarten, Caitlin W., additional, Oved, Joseph H., additional, Wray, Lisa, additional, Freedman, Jason L., additional, Wang, Yongping, additional, Kadauke, Stephan, additional, Hankins, Patricia, additional, Nicholas, Peter, additional, Worster, Elizabeth, additional, Kwiatkowski, Janet L., additional, Grupp, Stephan A., additional, Bunin, Nancy J., additional, and Olson, Timothy S., additional

Published
2024
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13. Unrelated and Haploidentical Peripheral Stem Cell Transplant with Tcrαβ/CD19 Depletion for the Treatment of Pediatric Patients with Inborn Errors of Immunity

Author

McClory, Susan E., primary, Elgarten, Caitlin W., additional, Quijano, Kaitlyn N, additional, Wohlschlaeger, Anne, additional, Hankins, Mrs. Patricia, additional, Wang, Lei, additional, Wang, Yongping, additional, Kadauke, Stephan, additional, Monos, Dimitri, additional, Olson, Timothy S., additional, Grupp, Stephan A., additional, Ochfeld, Elisa, additional, Heimall, Jennifer R., additional, and Bunin, Nancy J., additional

Published
2024
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14. Adenoviral Cellular Therapy with Adenovirus (ADV)-Specific Cytotoxic T-Lymphocytes (CTLs) for Resistant Infections in Children, Adolescent, and Young Adult (CAYA) Patients Following Allogeneic Hematopoietic Stem Cell Transplantation (AlloHSCT): A Promising Approach

Author

Schaefer, Edo, primary, Vu, Christine, additional, Flower, Allyson, additional, Ayello, Janet, additional, Chu, Yaya, additional, van de Ven, Carmella, additional, Mintzer, Elizabeth, additional, Harrison, Lauren, additional, Morris, Erin, additional, Wang, Yongping, additional, Shenoy, Shalini, additional, Lee, Dean Anthony, additional, Abu-Arja, Rolla, additional, Johnson, Bryon D, additional, Verneris, Michael R., additional, Dvorak, Christopher C., additional, Chu, Julia, additional, Cooke, Kenneth R., additional, Talano, Julie A, additional, Bunin, Nancy J., additional, and Cairo, Mitchell S., additional

Published
2024
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15. Phase II/III trial of a pre‐transplant farnesyl transferase inhibitor in juvenile myelomonocytic leukemia: A report from the Children's Oncology Group

Author

Stieglitz, Elliot, Ward, Ashley F, Gerbing, Robert B, Alonzo, Todd A, Arceci, Robert J, Liu, Y Lucy, Emanuel, Peter D, Widemann, Brigitte C, Cheng, Jennifer W, Jayaprakash, Nalini, Balis, Frank M, Castleberry, Robert P, Bunin, Nancy J, Loh, Mignon L, and Cooper, Todd M

Subjects
Biomedical and Clinical Sciences, Cardiovascular Medicine and Haematology, Oncology and Carcinogenesis, Clinical Trials and Supportive Activities, Rare Diseases, Pediatric, Pediatric Cancer, Transplantation, Cancer, Hematology, Clinical Research, Childhood Leukemia, Stem Cell Research, Evaluation of treatments and therapeutic interventions, 6.1 Pharmaceuticals, 6.2 Cellular and gene therapies, Adult, Aged, Aged, 80 and over, Antineoplastic Combined Chemotherapy Protocols, Child, Child, Preschool, Cytarabine, Disease-Free Survival, Enzyme Inhibitors, Farnesyl-Diphosphate Farnesyltransferase, Female, Hematopoietic Stem Cell Transplantation, Humans, Infant, Isotretinoin, Leukemia, Myelomonocytic, Juvenile, Male, Middle Aged, Quinolones, Survival Rate, Vidarabine, 13-cis retinoic acid, farnesyl transferase inhibitor, hematopoietic stem cell transplant, juvenile myelomonocytic leukemia, tipifarnib, Clinical Sciences, Paediatrics and Reproductive Medicine, Oncology & Carcinogenesis, Oncology and carcinogenesis, Paediatrics
Abstract

BackgroundJuvenile myelomonocytic leukemia (JMML) is not durably responsive to chemotherapy, and approximately 50% of patients relapse after hematopoietic stem cell transplant (HSCT). Here we report the activity and acute toxicity of the farnesyl transferase inhibitor tipifarnib, the response rate to 13-cis retinoic acid (CRA) in combination with cytoreductive chemotherapy, and survival following HSCT in children with JMML.ProcedureEighty-five patients with newly diagnosed JMML were enrolled on AAML0122 between 2001 and 2006. Forty-seven consented to receive tipifarnib in a phase II window before proceeding to a phase III trial of CRA in combination with fludarabine and cytarabine followed by HSCT and maintenance CRA. Thirty-eight patients enrolled only in the phase III trial.ResultsOverall response rate was 51% after tipifarnib and 68% after fludarabine/cytarabine/CRA. Tipifarnib did not increase pre-transplant toxicities. Forty-six percent of the 44 patients who received protocol compliant HSCT relapsed. Five-year overall survival was 55 ± 11% and event-free survival was 41 ± 11%, with no significant difference between patients who did or did not receive tipifarnib.ConclusionsAdministration of tipifarnib in the window setting followed by HSCT in patients with newly diagnosed JMML was safe and yielded a 51% initial response rate as a single agent, but failed to reduce relapse rates or improve long-term overall survival.

Published
2015

16. Correction to: Infections in Infants with SCID: Isolation, Infection Screening and Prophylaxis in PIDTC Centers

Author

Dorsey, Morna, Wright, Nicola A. M., Chaimowitz, Natalia S., Dávila Saldaña, Blachy J., Miller, Holly, Keller, Michael D., Thakar, Monica S., Shah, Ami J., Abu-Arja, Rolla, Andolina, Jeffrey, Aquino, Victor, Barnum, J. L., Bednarski, Jeffrey J., Bhatia, Monica, Bonilla, Francisco A., Butte, Manish J., Bunin, Nancy J., Burroughs, Lauri M., Chandra, Sharat, Chaudhury, Sonali, Chen, Karin, Chong, Hey, Cuvelier, Geoff, Dalal, Jignesh, DeFelice, Magee L., DeSantes, Kenneth B., Forbes, Lisa R., Gillio, Alfred, Goldman, Fred, Joshi, Avni Y., Kapoor, Neena, Knutsen, Alan P., Kobrynski, Lisa, Lieberman, Jay A., Leiding, Jennifer W., Oshrine, Benjamin, Patel, Kiran P., Prockop, Susan, Quigg, Troy C., Quinones, Ralph, Schultz, Kirk R., Seroogy, Christine, Shyr, David, Siegel, Subhadra, Smith, Angela R., Torgerson, Troy R., Vander Lugt, Mark T., Yu, Lolie C., Cowan, Morton J., Buckley, Rebecca H., Dvorak, Christopher C., Griffith, Linda M., Haddad, Elie, Kohn, Donald B., Logan, Brent, Notarangelo, Luigi D., Pai, Sung-Yun, Puck, Jennifer, Pulsipher, Michael A., and Heimall, Jennifer

Published
2021
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18. Germline CBL mutations cause developmental abnormalities and predispose to juvenile myelomonocytic leukemia.

Author

Niemeyer, Charlotte M, Kang, Michelle W, Shin, Danielle H, Furlan, Ingrid, Erlacher, Miriam, Bunin, Nancy J, Bunda, Severa, Finklestein, Jerry Z, Gorr, Thomas A, Mehta, Parinda, Schmid, Irene, Kropshofer, Gabriele, Corbacioglu, Selim, Lang, Peter J, Klein, Christoph, Schlegel, Paul-Gerhard, Heinzmann, Andrea, Schneider, Michaela, Starý, Jan, van den Heuvel-Eibrink, Marry M, Hasle, Henrik, Locatelli, Franco, Sakai, Debbie, Archambeault, Sophie, Chen, Leslie, Russell, Ryan C, Sybingco, Stephanie S, Ohh, Michael, Braun, Benjamin S, Flotho, Christian, and Loh, Mignon L

Subjects
Humans, Cryptorchidism, Genetic Predisposition to Disease, Pedigree, DNA Mutational Analysis, Developmental Disabilities, Germ-Line Mutation, Child, Preschool, Infant, Infant, Newborn, Female, Male, Proto-Oncogene Proteins c-cbl, Leukemia, Myelomonocytic, Juvenile, Child, Preschool, Newborn, Leukemia, Myelomonocytic, Juvenile, Developmental Biology, Biological Sciences, Medical and Health Sciences
Abstract

CBL encodes a member of the Cbl family of proteins, which functions as an E3 ubiquitin ligase. We describe a dominant developmental disorder resulting from germline missense CBL mutations, which is characterized by impaired growth, developmental delay, cryptorchidism and a predisposition to juvenile myelomonocytic leukemia (JMML). Some individuals experienced spontaneous regression of their JMML but developed vasculitis later in life. Importantly, JMML specimens from affected children show loss of the normal CBL allele through acquired isodisomy. Consistent with these genetic data, the common p.371Y>H altered Cbl protein induces cytokine-independent growth and constitutive phosphorylation of ERK, AKT and S6 only in hematopoietic cells in which normal Cbl expression is reduced by RNA interference. We conclude that germline CBL mutations have developmental, tumorigenic and functional consequences that resemble disorders that are caused by hyperactive Ras/Raf/MEK/ERK signaling and include neurofibromatosis type 1, Noonan syndrome, Costello syndrome, cardiofaciocutaneous syndrome and Legius syndrome.

Published
2010

19. Monogenic early-onset lymphoproliferation and autoimmunity: Natural history of STAT3 gain-of-function syndrome

Author

Leiding, Jennifer W., primary, Vogel, Tiphanie P., additional, Santarlas, Valentine G.J., additional, Mhaskar, Rahul, additional, Smith, Madison R., additional, Carisey, Alexandre, additional, Vargas-Hernández, Alexander, additional, Silva-Carmona, Manuel, additional, Heeg, Maximilian, additional, Rensing-Ehl, Anne, additional, Neven, Bénédicte, additional, Hadjadj, Jérôme, additional, Hambleton, Sophie, additional, Ronan Leahy, Timothy, additional, Meesilpavikai, Kornvalee, additional, Cunningham-Rundles, Charlotte, additional, Dutmer, Cullen M., additional, Sharapova, Svetlana O., additional, Taskinen, Mervi, additional, Chua, Ignatius, additional, Hague, Rosie, additional, Klemann, Christian, additional, Kostyuchenko, Larysa, additional, Morio, Tomohiro, additional, Thatayatikom, Akaluck, additional, Ozen, Ahmet, additional, Scherbina, Anna, additional, Bauer, Cindy S., additional, Flanagan, Sarah E., additional, Gambineri, Eleonora, additional, Giovannini-Chami, Lisa, additional, Heimall, Jennifer, additional, Sullivan, Kathleen E., additional, Allenspach, Eric, additional, Romberg, Neil, additional, Deane, Sean G., additional, Prince, Benjamin T., additional, Rose, Melissa J., additional, Bohnsack, John, additional, Mousallem, Talal, additional, Jesudas, Rohith, additional, Santos Vilela, Maria Marluce Dos, additional, O’Sullivan, Michael, additional, Pachlopnik Schmid, Jana, additional, Průhová, Štěpánka, additional, Klocperk, Adam, additional, Rees, Matthew, additional, Su, Helen, additional, Bahna, Sami, additional, Baris, Safa, additional, Bartnikas, Lisa M., additional, Chang Berger, Amy, additional, Briggs, Tracy A., additional, Brothers, Shannon, additional, Bundy, Vanessa, additional, Chan, Alice Y., additional, Chandrakasan, Shanmuganathan, additional, Christiansen, Mette, additional, Cole, Theresa, additional, Cook, Matthew C., additional, Desai, Mukesh M., additional, Fischer, Ute, additional, Fulcher, David A., additional, Gallo, Silvanna, additional, Gauthier, Amelie, additional, Gennery, Andrew R., additional, Gonçalo Marques, José, additional, Gottrand, Frédéric, additional, Grimbacher, Bodo, additional, Grunebaum, Eyal, additional, Haapaniemi, Emma, additional, Hämäläinen, Sari, additional, Heiskanen, Kaarina, additional, Heiskanen-Kosma, Tarja, additional, Hoffman, Hal M., additional, Gonzalez-Granado, Luis Ignacio, additional, Guerrerio, Anthony L., additional, Kainulainen, Leena, additional, Kumar, Ashish, additional, Lawrence, Monica G., additional, Levin, Carina, additional, Martelius, Timi, additional, Neth, Olaf, additional, Olbrich, Peter, additional, Palma, Alejandro, additional, Patel, Niraj C., additional, Pozos, Tamara, additional, Preece, Kahn, additional, Lugo Reyes, Saúl Oswaldo, additional, Russell, Mark A., additional, Schejter, Yael, additional, Seroogy, Christine, additional, Sinclair, Jan, additional, Skevofilax, Effie, additional, Suan, Daniel, additional, Suez, Daniel, additional, Szabolcs, Paul, additional, Velasco, Helena, additional, Warnatz, Klaus, additional, Walkovich, Kelly, additional, Worth, Austen, additional, Seppänen, Mikko R.J., additional, Torgerson, Troy R., additional, Sogkas, Georgios, additional, Ehl, Stephan, additional, Tangye, Stuart G., additional, Cooper, Megan A., additional, Milner, Joshua D., additional, Forbes Satter, Lisa R., additional, Aleshkevich, Svetlana, additional, Allende, Luis M., additional, Atkinson, T. Prescott, additional, Atschekzei, Faranaz, additional, Aydemir, Sezin, additional, Aygunes, Utku, additional, Barlogis, Vincent, additional, Baumann, Ulrich, additional, Belko, John, additional, Bezrodnik, Liliana, additional, Biebl, Ariane, additional, Broderick, Lori, additional, Bunin, Nancy J., additional, Caldirola, Maria Soledad, additional, Castelle, Martin, additional, Celmeli, Fatih, additional, Charbonnier, Louis-Marie, additional, Chatila, Talal A., additional, Chellapandian, Deepak, additional, Cokugras, Haluk, additional, Conlon, Niall, additional, Cox, Fionnuala, additional, Crickx, Etienne, additional, Dalgic, Buket, additional, ASH Dalm, Virgil, additional, Danielian, Silvia, additional, Dominguez-Pinilla, Nerea, additional, Dujovny, Tal, additional, Ebbo, Mikael, additional, Eken, Ahmet, additional, Esty, Brittany, additional, Fabre, Alexandre, additional, Fischer, Alain, additional, Hannibal, Mark, additional, Huppert, Laura, additional, Ikeda, Marc D., additional, Jolles, Stephen, additional, Jolly, Kent W., additional, Jones, Neil, additional, Kanariou, Maria, additional, Karakoc-Aydiner, Elif, additional, Karamantziani, Theoni, additional, Kelaidi, Charikleia, additional, Keogan, Mary, additional, Pac Kisaarslan, Ayşenur, additional, Kiykim, Ayca, additional, Kotsonis, Kosmas, additional, Kuzmenko, Natalia, additional, Leroy, Sylvie, additional, Lianou, Dimitra, additional, Longhurst, Hilary, additional, Lorenz, Myriam Ricarda, additional, Maffucci, Patrick, additional, Manson, Ania, additional, Marchal, Sarah, additional, Malphettes, Marion, additional, Marega, Lia Furlaneto, additional, Mauracher, Andrea A., additional, Miller, Holly, additional, Mombourquette, Joy, additional, Morgan, Noel G., additional, Mukhina, Anna, additional, Nathalie, Aladjidi, additional, Nelken, Brigitte, additional, Nolan, David, additional, Norlin, Anna-Carin, additional, Oleastro, Matias, additional, Ozcan, Alper, additional, Pasquet, Marlene, additional, Pegler, José Roberto, additional, Picard, Capucine, additional, Polychronopoulou, Sophia, additional, Quartier, Pierre, additional, Quesada, Juan Francisco, additional, Ramakers, Jan, additional, Randall, Katrina L., additional, Rao, V. Koneti, additional, Remiker, Allison, additional, Resin, Geraldine, additional, Richmond, Peter, additional, Rieux-Laucat, Frederic, additional, Rodina, Yulia, additional, Rohrlich, Pierre, additional, Sachs, Johnathan, additional, Sakovich, Inga, additional, Santarlas, Christopher, additional, Sari, Sinan, additional, Sawicki, Gregory, additional, Schauer, Uwe, additional, Scheffler Mendoza, Selma C., additional, Schvetz, Oksana, additional, Schmidt, Reinhold Ernst, additional, Schwarz, Klaus, additional, Sediva, Anna, additional, Sinclair, Kyle, additional, Slatter, Mary, additional, Sleasman, John, additional, Stergiou, Katerina, additional, Suratannon, Narissara, additional, Tanita, Kay, additional, Thompson, Grace, additional, Travis, Stephen, additional, Trojan, Timothy, additional, Tsinti, Maria, additional, Unal, Ekrem, additional, Urdinez, Luciano, additional, Vazquez-Gomez, Felisa, additional, Villa, Mariana, additional, Weinrich, Michael, additional, Weiss, Mitchell J., additional, Wright, Benjamin, additional, Yilmaz, Ebru, additional, Zachova, Radana, additional, and Zhang, Yu, additional

Published
2023
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21. Safety and Efficacy of Virus-Specific Cytotoxic T-Lymphocytes Manufactured By the IFN-γ Cytokine Capture System for the Treatment of Refractory Adenovirus, Cytomegalovirus, Epstein Barr Virus, and BK Virus Infections in Children, Adolescents and Young Adults after Allogeneic Hematopoietic Stem Cell Transplantation, Solid Organ Transplantation, or with Primary Immunodeficiency (IND# 17449)

Author

Flower, Allyson, primary, Talano, Julie, additional, Ayello, Janet, additional, Rigot, Olivia, additional, Harrison, Lauren, additional, Morris, Erin, additional, Mintzer, Elizabeth, additional, Maryamchick, Elena, additional, Wang, Yongping, additional, O’Donnell, Lynn C., additional, Abu-Arja, Rolla, additional, Lee, Dean A., additional, Johnson, Bryon D, additional, Chu, Julia, additional, Dvorak, Christopher C., additional, Shenoy, Shalini, additional, Bunin, Nancy J., additional, and Cairo, Mitchell S., additional

Published
2023
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22. Allogenic Hematopoietic Cell Transplantations Are Effective in Patients with p47phox Chronic Granulomatous Disease: A Primary Immune Deficiency Treatment Consortium Study

Author

Grunebaum, Eyal, primary, Arnold, Danielle E., additional, Logan, Brent, additional, Parikh, Suhag, additional, Marsh, Rebecca A., additional, Griffith, Linda M., additional, Mallhi, Kanwaldeep, additional, Chellapandian, Deepak, additional, Lim, Stephanie Si, additional, Deal, Christin L., additional, Murguía-Favela, Luis, additional, Mousallem, Talal I., additional, Prasad, Prof. Vinod K., additional, Teira, Pierre, additional, Touzot, Fabien, additional, Bunin, Nancy J., additional, Heimall, Jennifer R., additional, Burroughs, Lauri M., additional, Kapadia, Malika, additional, Prockop, Susan, additional, Chandra, Sharat, additional, Chandrakasan, Shanmuganathan, additional, Chaudhury, Sonali, additional, Broglie, Larisa, additional, O’Reilly, Richard J., additional, Dávila Saldaña, Blachy J., additional, Schaefer, Edo, additional, Chong, Hey, additional, Bednarski, Jeffrey J., additional, Rayes, Ahmad, additional, DeSantes, Kenneth, additional, Kohn, Donald B., additional, Notarangelo, Luigi D., additional, Pai, Sung-Yun, additional, Puck, Jennifer, additional, Torgerson, Troy, additional, Cowan, Morton J., additional, Dvorak, Christopher C., additional, Satter, Lisa Forbes, additional, Haddad, Elie, additional, Pulsipher, Michael, additional, Malech, Harry L., additional, Kang, Elizabeth M., additional, and Leiding, Jennifer W., additional

Published
2023
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23. Manufacture, Immunological Characterization and Clinical Response of GMP Grade Sars-COV-2 Cytotoxic T Lymphocytes (CTLs) Utilizing the Clinimacs® Cytokine Capture System

Author

Chu, Yaya, primary, Lamb, Margaret, additional, Maryamchik, Elena, additional, Rigot, Olivia, additional, Ayello, Janet, additional, Harrison, Lauren, additional, Mintzer, Elizabeth, additional, Shaw, Rosemarie, additional, Behbehani, Gregory, additional, Mardis, Elaine, additional, Miller, Katherine, additional, Rao Venkata, Lakshmi Prakruthi, additional, Chang, Hsiaochi, additional, Lee, Dean A., additional, Rosenthal, Elana Y., additional, Kadauke, Stephan, additional, Bunin, Nancy J., additional, Talano, Julie, additional, Johnson, Bryon D, additional, Wang, Yongping, additional, and Cairo, Mitchell S., additional

Published
2023
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24. Updated Efficacy and Safety of Tabelecleucel in Patients with Epstein-Barr Virus-Positive (EBV+) Leiomyosarcomas (LMS)

Author

Jiménez-Kurlander, Lauren S, primary, Behr, Gerald, additional, Bunin, Nancy J, additional, Doubrovina, Ekaterina, additional, Dinavahi, Rajani, additional, Gamelin, Laurence, additional, Ghobadi, Armin, additional, Mascarenhas, Leo, additional, Nikiforow, Sarah, additional, O'Reilly, Richard J, additional, Price, Anita, additional, Shulkin, Barry, additional, Srinivasan, Ashok, additional, Suser, Stephanie, additional, Wahlstrom, Justin, additional, Xing, Baodong, additional, and Prockop, Susan, additional

Published
2022
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25. 125 - Hematopoietic Cell Transplantation in 240 Patients with Chronic Granulomatous Disease: A Pidtc Report

Author

Arnold, Danielle E., Parikh, Suhag, Logan, Brent, Marsh, Rebecca, Griffith, Linda M., Wu, Ruizhe, Mallhi, Kanwaldeep, Chellapandian, Deepak, Si, Stephanie, Grunebaum, Eyal, Broglie, Larisa, Prasad, Vinod K., Heimall, Jennifer, Bunin, Nancy J., Teira, Pierre, Touzot, Fabien, Burroughs, Lauri M., Petrovic, Aleksandra, Bleesing, Jack J., Chandra, Sharat, Kapoor, Neena, Dara, Jasmeen, Dorsey, Morna, Williams, Olatundun, Kapadia, Malika, Bednarski, Jeffrey, Rayes, Ahmad, Chen, Karin, Chong, Hey, Cuvelier, Geoff D.E., Forbes, Lisa R., Martinez, Caridad, Vander Lugt, Mark T., Yu, Lolie C., Chandrakasan, Shanmuganathan, Joshi, Avni, Prockop, Susan E., Davila Saldana, Blachy J., Aquino, Victor, Ebens, Christen L., Madden, Lisa, DeSantes, Kenneth, Milner, Jordan, Rangarajan, Hemalatha G., Shah, Ami J, Gillio, Alfred P., Knutsen, Alan P., Miller, Holly K., Moore, Theodore B., Wright, Nicola, Cowan, Morton J., Dvorak, Christopher C., Haddad, Elie, Kohn, Donald B., Notarangelo, Luigi D., Pai, Sung-Yun, Puck, Jennifer, Pulsipher, Mike A., Torgerson, Troy, Malech, Harry L., Kang, Elizabeth M., and Leiding, Jennifer W.

Published
2022
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26. 3 - Comparison of Outcomes in Patients with Cerebral Adrenoleukodystrophy (CALD) Receiving Elivaldogene Autotemcel (eli-cel; Lenti-D) Gene Therapy in Clinical Trials Versus Those Receiving Allogeneic Hematopoietic Stem Cell Transplant in a Contemporaneous Comparator Study

Author

Duncan, Christine N., Kühl, Jörn-Sven, Chiesa, Robert, Boelens, Jaap-Jan, Eichler, Florian, Sevin, Caroline, Dalle, Jean-Hugues, De Oliveira, Satiro N., Amartino, Hernan M., Kapoor, Neena, Prasad, Vinod K., Jones, Simon, Algeri, Mattia, Bunin, Nancy J., Diaz-de-Heredia, Cristina, Thrasher, Adrian J., Fernandes, Juliana Folloni, Smith, Nicholas, Shah, Ami J, Locatelli, Franco, Engelen, Marc, Lindemans, Caroline A., Dietz, Andrew C., Pan, Lin, Sieker, Jakob, Williams, David A., and Orchard, Paul J.

Published
2022
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27. Hematopoietic Cell Transplantation in 240 Patients with Chronic Granulomatous Disease: A Pidtc Report

Author

Arnold, Danielle E., primary, Parikh, Suhag, additional, Logan, Brent, additional, Marsh, Rebecca, additional, Griffith, Linda M., additional, Wu, Ruizhe, additional, Mallhi, Kanwaldeep, additional, Chellapandian, Deepak, additional, Si, Stephanie, additional, Grunebaum, Eyal, additional, Broglie, Larisa, additional, Prasad, Vinod K., additional, Heimall, Jennifer, additional, Bunin, Nancy J., additional, Teira, Pierre, additional, Touzot, Fabien, additional, Burroughs, Lauri M., additional, Petrovic, Aleksandra, additional, Bleesing, Jack J., additional, Chandra, Sharat, additional, Kapoor, Neena, additional, Dara, Jasmeen, additional, Dorsey, Morna, additional, Williams, Olatundun, additional, Kapadia, Malika, additional, Bednarski, Jeffrey, additional, Rayes, Ahmad, additional, Chen, Karin, additional, Chong, Hey, additional, Cuvelier, Geoff D.E., additional, Forbes, Lisa R., additional, Martinez, Caridad, additional, Vander Lugt, Mark T., additional, Yu, Lolie C., additional, Chandrakasan, Shanmuganathan, additional, Joshi, Avni, additional, Prockop, Susan E., additional, Davila Saldana, Blachy J., additional, Aquino, Victor, additional, Ebens, Christen L., additional, Madden, Lisa, additional, DeSantes, Kenneth, additional, Milner, Jordan, additional, Rangarajan, Hemalatha G., additional, Shah, Ami J, additional, Gillio, Alfred P., additional, Knutsen, Alan P., additional, Miller, Holly K., additional, Moore, Theodore B., additional, Wright, Nicola, additional, Cowan, Morton J., additional, Dvorak, Christopher C., additional, Haddad, Elie, additional, Kohn, Donald B., additional, Notarangelo, Luigi D., additional, Pai, Sung-Yun, additional, Puck, Jennifer, additional, Pulsipher, Mike A., additional, Torgerson, Troy, additional, Malech, Harry L., additional, Kang, Elizabeth M., additional, and Leiding, Jennifer W., additional

Published
2022
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28. Feasibility, Safety and Efficacy of Haploidentical Viral Cytotoxic T-Lymphocytes for Children, Adolescents, and Young Adults (CAYA) with Refractory Viral Infections in Primary Immunodeficiencies (PID) Post Solid Organ Transplant (SOT) or Allogeneic Hematopoietic Stem Cell Transplantation (AlloHSCT)

Author

Milner, Jordan, primary, Talano, Julie-An, additional, Harrison, Lauren, additional, Ayello, Janet, additional, Flower, Allyson, additional, Chu, Yaya, additional, Rigot, Olivia, additional, Johnson, Bryon D, additional, Li, Yimei, additional, Lee, Dean Anthony, additional, Abu-Arja, Rolla, additional, Chu, Julia, additional, Dvorak, Christopher C., additional, O’Donnell, Lynn C., additional, Wang, Yongping, additional, Bunin, Nancy J., additional, and Cairo, Mitch S., additional

Published
2022
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29. Comparison of Outcomes in Patients with Cerebral Adrenoleukodystrophy (CALD) Receiving Elivaldogene Autotemcel (eli-cel; Lenti-D) Gene Therapy in Clinical Trials Versus Those Receiving Allogeneic Hematopoietic Stem Cell Transplant in a Contemporaneous Comparator Study

Author

Duncan, Christine N., primary, Kühl, Jörn-Sven, additional, Chiesa, Robert, additional, Boelens, Jaap-Jan, additional, Eichler, Florian, additional, Sevin, Caroline, additional, Dalle, Jean-Hugues, additional, De Oliveira, Satiro N., additional, Amartino, Hernan M., additional, Kapoor, Neena, additional, Prasad, Vinod K., additional, Jones, Simon, additional, Algeri, Mattia, additional, Bunin, Nancy J., additional, Diaz-de-Heredia, Cristina, additional, Thrasher, Adrian J., additional, Fernandes, Juliana Folloni, additional, Smith, Nicholas, additional, Shah, Ami J, additional, Locatelli, Franco, additional, Engelen, Marc, additional, Lindemans, Caroline A., additional, Dietz, Andrew C., additional, Pan, Lin, additional, Sieker, Jakob, additional, Williams, David A., additional, and Orchard, Paul J., additional

Published
2022
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30. Outcome of Myeloablative Conditioning and Unrelated Donor Hematopoietic Cell Transplantation for Childhood Acute Lymphoblastic Leukemia in Third Remission

Author

Nemecek, Eneida R., Ellis, Kristin, He, Wensheng, Bunin, Nancy J., Bajwa, Rajinder S., Cheerva, Alexandra, Cairo, Mitchell S., Dvorak, Christopher, Duval, Michel, Davies, Stella, Eapen, Mary, Gross, Thomas G., Hussein, Ayad A., MacMillan, Margaret L., Mehta, Parinda A., Pulsipher, Michael A., Seber, Adriana, Woolfrey, Ann E., Frangoul, Haydar A., and Carpenter, Paul A.

Published
2011
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32. Reduced-Intensity/Reduced-Toxicity Conditioning Approaches Are Tolerated in XIAP Deficiency but Patients Fare Poorly with Acute GVHD

Author

Arnold, Danielle E., primary, Nofal, Rofida, additional, Wakefield, Connor, additional, Lehmberg, Kai, additional, Wustrau, Katharina, additional, Albert, Michael H., additional, Morris, Emma C., additional, Heimall, Jennifer R., additional, Bunin, Nancy J., additional, Kumar, Ashish, additional, Jordan, Michael B., additional, Cole, Theresa, additional, Choo, Sharon, additional, Brettig, Tim, additional, Speckmann, Carsten, additional, Ehl, Stephan, additional, Salamonowicz, Malgorzata, additional, Wahlstrom, Justin, additional, Rao, Kanchan, additional, Booth, Claire, additional, Worth, Austen, additional, and Marsh, Rebecca A., additional

Published
2021
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34. Chimeric Antigen Receptor T Cells for Sustained Remissions in Leukemia

Author

Maude, Shannon L., Frey, Noelle, Shaw, Pamela A., Aplenc, Richard, Barrett, David M., Bunin, Nancy J., Chew, Anne, Gonzalez, Vanessa E., Zheng, Zhaohui, Lacey, Simon F., Mahnke, Yolanda D., Melenhorst, Jan J., Rheingold, Susan R., Shen, Angela, Teachey, David T., Levine, Bruce L., June, Carl H., Porter, David L., and Grupp, Stephan A.

Published
2014
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35. Outcomes of Allogeneic Hematopoietic Stem Cell Transplantation in Patients with Cerebral Adrenoleukodystrophy: Effects of Donor Cell Source and Match, Conditioning Regimen, and Stage of Cerebral Disease

Author

Boelens, Jaap Jan, primary, Chiesa, Robert, additional, Duncan, Christine N., additional, Kühl, Jörn-Sven, additional, Sevin, Caroline, additional, Kapoor, Neena, additional, Prasad, Vinod K., additional, Lindemans, Caroline A., additional, Jones, Simon A., additional, Amartino, Hernan M., additional, Algeri, Mattia, additional, Bunin, Nancy J., additional, Diaz-de-Heredia, Cristina, additional, Shamir, Esther, additional, Timm, Alison, additional, Dietz, Andrew C., additional, McNeil, Elizabeth, additional, and Orchard, Paul J., additional

Published
2021
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36. Safety and Efficacy of GMP Clinical Grade Manufacturing of Virus Specific Cytotoxic T Lymphocytes (vCTLS) Against Cytomegalovirus (CMV), Epstein Barr Virus ( EBV), Adenovirus (ADV) and BKV to Treat Medically Refractory Viral Infections Post Allogeneic Hematopoietic Stem Cell Transplantation (AlloHSCT) in Children, Adolescents and Young Adults (CAYA) (IND# 17449)

Author

Ayello, Janet, primary, Flower, Allyson, additional, Kuhns, Robert, additional, Colon, Lin Marie, additional, Fallon, Bernadette, additional, Rigot, Olivia, additional, Shaw, Rosemarie, additional, Harrison, Lauren, additional, Morris, Erin, additional, Sturhahn, Meghan, additional, Gonzalez, Isabel D, additional, Wang, Yongping, additional, O’Donnell, Lynn C., additional, Lee, Dean Anthony, additional, Talano, Julie-An, additional, Johnson, Bryon D, additional, Bunin, Nancy J., additional, and Cairo, Mitchell S., additional

Published
2021
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38. High OS, PFS and EFS with Low Rates of Rejection and GVHD after KIR-Favorable abCD3/CD19 Depleted Haploidentical HCT in Children with ALL/AML/MDS: Primary Analysis of the Pediatric Transplantation and Cellular Therapy Consortium ONC1401 Trial

Author

Abdel-Azim, Hisham, primary, Dvorak, Christopher C, additional, Bunin, Nancy J., additional, Lalefar, Nahal, additional, Anderson, Eric, additional, Flower, Allyson, additional, Talano, Julie-An, additional, Ahn, Kwang Woo, additional, Chaudhury, Sonali, additional, Kitko, Carrie Lynn, additional, Monos, Dimitri, additional, Wing, Leung, additional, and Pulsipher, Mike A., additional

Published
2021
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40. Correction to: Infections in Infants with SCID: Isolation, Infection Screening and Prophylaxis in PIDTC Centers

Author

Dorsey, Morna, primary, Wright, Nicola A. M., additional, Chaimowitz, Natalia S., additional, Dávila Saldaña, Blachy J., additional, Miller, Holly, additional, Keller, Michael D., additional, Thakar, Monica S., additional, Shah, Ami J., additional, Abu-Arja, Rolla, additional, Andolina, Jeffrey, additional, Aquino, Victor, additional, Barnum, J. L., additional, Bednarski, Jeffrey J., additional, Bhatia, Monica, additional, Bonilla, Francisco A., additional, Butte, Manish J., additional, Bunin, Nancy J., additional, Burroughs, Lauri M., additional, Chandra, Sharat, additional, Chaudhury, Sonali, additional, Chen, Karin, additional, Chong, Hey, additional, Cuvelier, Geoff, additional, Dalal, Jignesh, additional, DeFelice, Magee L., additional, DeSantes, Kenneth B., additional, Forbes, Lisa R., additional, Gillio, Alfred, additional, Goldman, Fred, additional, Joshi, Avni Y., additional, Kapoor, Neena, additional, Knutsen, Alan P., additional, Kobrynski, Lisa, additional, Lieberman, Jay A., additional, Leiding, Jennifer W., additional, Oshrine, Benjamin, additional, Patel, Kiran P., additional, Prockop, Susan, additional, Quigg, Troy C., additional, Quinones, Ralph, additional, Schultz, Kirk R., additional, Seroogy, Christine, additional, Shyr, David, additional, Siegel, Subhadra, additional, Smith, Angela R., additional, Torgerson, Troy R., additional, Vander Lugt, Mark T., additional, Yu, Lolie C., additional, Cowan, Morton J., additional, Buckley, Rebecca H., additional, Dvorak, Christopher C., additional, Griffith, Linda M., additional, Haddad, Elie, additional, Kohn, Donald B., additional, Logan, Brent, additional, Notarangelo, Luigi D., additional, Pai, Sung-Yun, additional, Puck, Jennifer, additional, Pulsipher, Michael A., additional, and Heimall, Jennifer, additional

Published
2020
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43. 423 - Manufacture, Immunological Characterization and Clinical Response of GMP Grade Sars-COV-2 Cytotoxic T Lymphocytes (CTLs) Utilizing the Clinimacs® Cytokine Capture System

Author

Chu, Yaya, Lamb, Margaret, Maryamchik, Elena, Rigot, Olivia, Ayello, Janet, Harrison, Lauren, Mintzer, Elizabeth, Shaw, Rosemarie, Behbehani, Gregory, Mardis, Elaine, Miller, Katherine, Rao Venkata, Lakshmi Prakruthi, Chang, Hsiaochi, Lee, Dean A., Rosenthal, Elana Y., Kadauke, Stephan, Bunin, Nancy J., Talano, Julie, Johnson, Bryon D, Wang, Yongping, and Cairo, Mitchell S.

Published
2023
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44. 424 - Safety and Efficacy of Virus-Specific Cytotoxic T-Lymphocytes Manufactured By the IFN-γ Cytokine Capture System for the Treatment of Refractory Adenovirus, Cytomegalovirus, Epstein Barr Virus, and BK Virus Infections in Children, Adolescents and Young Adults after Allogeneic Hematopoietic Stem Cell Transplantation, Solid Organ Transplantation, or with Primary Immunodeficiency (IND# 17449)

Author

Flower, Allyson, Talano, Julie, Ayello, Janet, Rigot, Olivia, Harrison, Lauren, Morris, Erin, Mintzer, Elizabeth, Maryamchick, Elena, Wang, Yongping, O’Donnell, Lynn C., Abu-Arja, Rolla, Lee, Dean A., Johnson, Bryon D, Chu, Julia, Dvorak, Christopher C., Shenoy, Shalini, Bunin, Nancy J., and Cairo, Mitchell S.

Published
2023
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45. 382 - Allogenic Hematopoietic Cell Transplantations Are Effective in Patients with p47phox Chronic Granulomatous Disease: A Primary Immune Deficiency Treatment Consortium Study

Author

Grunebaum, Eyal, Arnold, Danielle E., Logan, Brent, Parikh, Suhag, Marsh, Rebecca A., Griffith, Linda M., Mallhi, Kanwaldeep, Chellapandian, Deepak, Lim, Stephanie Si, Deal, Christin L., Murguía-Favela, Luis, Mousallem, Talal I., Prasad, Prof. Vinod K., Teira, Pierre, Touzot, Fabien, Bunin, Nancy J., Heimall, Jennifer R., Burroughs, Lauri M., Kapadia, Malika, Prockop, Susan, Chandra, Sharat, Chandrakasan, Shanmuganathan, Chaudhury, Sonali, Broglie, Larisa, O’Reilly, Richard J., Dávila Saldaña, Blachy J., Schaefer, Edo, Chong, Hey, Bednarski, Jeffrey J., II, Rayes, Ahmad, DeSantes, Kenneth, Kohn, Donald B., Notarangelo, Luigi D., Pai, Sung-Yun, Puck, Jennifer, Torgerson, Troy, Cowan, Morton J., Dvorak, Christopher C., Satter, Lisa Forbes, Haddad, Elie, Pulsipher, Michael, Malech, Harry L., Kang, Elizabeth M., and Leiding, Jennifer W.

Published
2023
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47. Infections in Infants with SCID: Isolation, Infection Screening, and Prophylaxis in PIDTC Centers

Author

Dorsey, Morna J., primary, Wright, Nicola A. M., additional, Chaimowitz, Natalia S., additional, Dávila Saldaña, Blachy J., additional, Miller, Holly, additional, Keller, Michael D., additional, Thakar, Monica S., additional, Shah, Ami J., additional, Abu-Arja, Rolla, additional, Andolina, Jeffrey, additional, Aquino, Victor, additional, Barnum, J. L., additional, Bednarski, Jeffrey J., additional, Bhatia, Monica, additional, Bonilla, Francisco A., additional, Butte, Manish J., additional, Bunin, Nancy J., additional, Chandra, Sharat, additional, Chaudhury, Sonali, additional, Chen, Karin, additional, Chong, Hey, additional, Cuvelier, Geoffrey D. E., additional, Dalal, Jignesh, additional, DeFelice, Magee L., additional, DeSantes, Kenneth B., additional, Forbes, Lisa R., additional, Gillio, Alfred, additional, Goldman, Fred, additional, Joshi, Avni Y., additional, Kapoor, Neena, additional, Knutsen, Alan P., additional, Kobrynski, Lisa, additional, Lieberman, Jay A., additional, Leiding, Jennifer W., additional, Oshrine, Benjamin, additional, Patel, Kiran P., additional, Prockop, Susan, additional, Quigg, Troy C., additional, Quinones, Ralph, additional, Schultz, Kirk R., additional, Seroogy, Christine, additional, Shyr, David, additional, Siegel, Subhadra, additional, Smith, Angela R., additional, Torgerson, Troy R., additional, Vander Lugt, Mark T., additional, Yu, Lolie C., additional, Cowan, Morton J., additional, Buckley, Rebecca H., additional, Dvorak, Christopher C., additional, Griffith, Linda M., additional, Haddad, Elie, additional, Kohn, Donald B., additional, Logan, Brent, additional, Notarangelo, Luigi D., additional, Pai, Sung-Yun, additional, Puck, Jennifer, additional, Pulsipher, Michael A., additional, and Heimall, Jennifer, additional

Published
2020
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48. 131 - Feasibility, Safety and Efficacy of Haploidentical Viral Cytotoxic T-Lymphocytes for Children, Adolescents, and Young Adults (CAYA) with Refractory Viral Infections in Primary Immunodeficiencies (PID) Post Solid Organ Transplant (SOT) or Allogeneic Hematopoietic Stem Cell Transplantation (AlloHSCT)

Author

Milner, Jordan, Talano, Julie-An, Harrison, Lauren, Ayello, Janet, Flower, Allyson, Chu, Yaya, Rigot, Olivia, Johnson, Bryon D, Li, Yimei, Lee, Dean Anthony, Abu-Arja, Rolla, Chu, Julia, Dvorak, Christopher C., O’Donnell, Lynn C., Wang, Yongping, Bunin, Nancy J., and Cairo, Mitch S.

Published
2022
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49. 883 – Integration of Clinical, Immunologic and Genomic Data in Children with Very-Early Onset Inflammatory Bowel Disease Leads to Identification of Candidates for Curative Therapy with Hematopoeitic Stem Cell Transplantation

Author

Patel, Trusha, primary, Conrad, Maire A., additional, Shraim, Rawan, additional, Merz, Audrey, additional, Maurer, Kelly, additional, Dawany, Noor, additional, Devoto, Marcella, additional, Behrens, Edward, additional, Bunin, Nancy J., additional, Freedman, Jason L., additional, Giraudo, Claudio, additional, Sullivan, Kathleen E., additional, and Kelsen, Judith R., additional

Published
2019
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50. Rapid Manufacture and Isolation of Highly Viable Interferon-γ Expressing Virus-Specific T Cells: Validation Results for the Virus-Specific Cytotoxic T-Lymphocyte Consortium for Refractory EBV, CMV, and Adenovirus Infections

Author

Kadauke, Stephan, primary, Johnson, Bryon D., additional, Harrison, Lauren, additional, O'Donnell, Lynn C., additional, Wang, Youwei, additional, Ayello, Janet, additional, Talano, Julie-An M., additional, Lee, Dean, additional, Bunin, Nancy J., additional, Wang, Yongping, additional, and Cairo, Mitchell S., additional

Published
2019
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